Poly(beta-amino ester)s as gene delivery vehicles: challenges and opportunities.
Poly(beta-amino ester)s as gene delivery vehicles: challenges and opportunities.
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DOI：
10.1080/17425247.2020.1796628
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发表时间：
2020-10
影响因子：
6.6
通讯作者：
Tzeng SY
中科院分区：
文献类型：
--
作者：
Karlsson J;Rhodes KR;Green JJ;Tzeng SY
基因递送技术正在被开发用于越来越多的生物医学应用，递送载体包括病毒和非病毒材料。在用于非病毒基因递送的生物材料中，聚（β-氨基酯）（PBAE），一类合成的、可生物降解的聚合物，已经上升为已用于多种体外和体内应用的领先基因递送载体。本文综述了PBAE的主要特性及其发展，包括讨论PBAE用于基因递送应用的优点和缺点。使用PBAE，以改善其他药物载体的性能也进行了总结。PBAE被设计为具有理想的基因递送的多种特性，包括它们的可逆正电荷，其促进与核酸的结合以及赋予高缓冲能力，以及它们在温和条件下的快速降解性。同时，它们的一些性质也导致纳米颗粒在生理环境中的不稳定性和低转染效率。然而，PBAE可以容易地进行化学改性以及与其他材料非共价共混，这使得它们可以专门定制，以克服各种应用的输送障碍。
Gene delivery technologies are being developed for an increasing number of biomedical applications, with delivery vehicles including viruses and non-viral materials. Among biomaterials used for non-viral gene delivery, poly(beta-amino ester)s (PBAEs), a class of synthetic, biodegradable polymers, have risen as a leading gene delivery vehicle that has been used for multiple applications in vitro and in vivo. This review summarizes the key properties of PBAEs and their development, including a discussion of the advantages and disadvantages of PBAEs for gene delivery applications. The use of PBAEs to improve the properties of other drug delivery vehicles is also summarized. PBAEs are designed to have multiple characteristics that are ideal for gene delivery, including their reversible positive charge, which promotes binding to nucleic acids as well as imparting high buffering capacity, and their rapid degradability under mild conditions. Simultaneously, some of their properties also lead to nanoparticle instability and low transfection efficiency in physiological environments. The ease with which PBAEs can be chemically modified as well as non-covalently blended with other materials, however, allows them to be customized specifically to overcome delivery barriers for varied applications.
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